This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Study of Parathyroid Hormone Following Sequential Cord Blood Transplantation From an Unrelated Donor (NCT00393380), a na phase 2 study. The primary outcome is Median Time to Neutrophil Engraftment (Defined as an Absolute Neutrophil Count [ANC] Greater Than 500).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00393380 |
| Phase | Phase 2 |
| Condition | Leukemia, Myeloid, Chronic, Anemia, Aplastic, Myelofibrosis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 13 |
| Arms in the primary outcome | n/a |
| Lead sponsor | The Emmes Company, LLC |
| Results posted | Yes |
2. Clinical Question
Population
The registry record says 13 patients received the transplant. The listed conditions include several leukemias, myelofibrosis, lymphoma, Hodgkin disease, and aplastic anemia. The record does not give further detail about participant characteristics or how many participants had each condition.
Intervention
Parathyroid hormone, also identified as teriparatide, was given after a double umbilical cord blood transplant. The study summary describes the transplant context as sequential cord blood transplantation from an unrelated donor.
Comparator
No comparison group is listed. The study is recorded as a single-group study, with allocation listed as not applicable and no masking. The record therefore does not provide a like-for-like control group for assessing outcomes.
Primary question
The study aimed to determine whether adding parathyroid hormone after transplantation would improve engraftment. Its primary endpoint was median time to neutrophil engraftment, defined as an absolute neutrophil count (ANC) greater than 500. The result for this endpoint is not reported in the registry record.
Engraftment describes the transplanted stem cells beginning to grow and produce new blood cells, as explained in the registry summary. The primary question is thus about the timing of a specified blood-count milestone after transplantation, rather than a direct comparison of outcomes between treatment groups. Because only a single group is recorded, any posted outcome would describe the participants who received the intervention in this study; it would not, by itself, establish what would have happened to comparable participants without parathyroid hormone.
The registry lists additional outcomes, including graft-versus-host disease, platelet engraftment, transplant-related mortality, relapse, overall survival, and disease-free survival. Their result values are not reported in the registry record. The record also does not state why the study was terminated, or whether termination was related to recruitment, safety, or another consideration.
3. Trial Design
This is a Phase II, single-group study of parathyroid hormone (teriparatide) following myeloablative sequential unrelated cord blood transplantation. The registry describes the treatment as parathyroid hormone after double umbilical cord blood transplant. It records 13 patients as having received the transplant. Allocation is listed as not applicable, and masking is listed as none. There is no concurrent control group described, so the design is not a randomized comparison.
The registry gives a start date of 2006-09 and a primary completion date of 2009-11. Its current status is terminated. The reason for termination is not reported in the registry record. The record identifies The Emmes Company, LLC as sponsor. It does not report a detailed protocol description, treatment schedule, dose, duration, eligibility criteria, or sample-size justification.
The primary endpoint is median time to neutrophil engraftment, defined by ANC greater than 500. The timeframe says the statistic is calculated at Day 42, while ANC counts are measured daily up through discharge. The listed dispersion is the full range. The registry does not include the median or range values in the registry record for this analysis.
Secondary outcomes listed include acute graft-versus-host disease grades II-IV at Day 100, chronic graft-versus-host disease, platelet engraftment defined as greater than 20,000, 100-day transplant-related mortality, relapse, overall survival, and disease-free survival. Results for these outcomes are not reported in the registry record. The record also does not describe the analysis population for each endpoint, how missing observations were handled, or whether any interim analyses took place.
These design details set limits on interpretation. A single-group study can describe observed outcomes after the intervention, but without a control group it cannot isolate the intervention’s effect from other influences. The registry information does not support a causal comparison or a claim that the intervention changed engraftment.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: Thirteen patients who were enrolled proceeded to the cord blood transplant.
Overall Study
| Stage | Parathyroid Hormone (Teriparatide) |
|---|---|
| STARTED | 13 |
| COMPLETED | 13 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: 13 patients received the transplant.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Parathyroid Hormone (Teriparatide) |
|---|---|---|
| Participants analysed | 13 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 11 |
| Age, Categorical: >=65 years | Participants | 2 |
| Age Continuous | years | 39 (25 to 67) |
| Sex: Female, Male: Female | Participants | 6 |
| Sex: Female, Male: Male | Participants | 7 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 10 |
| Race (NIH/OMB): More than one race | Participants | 2 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 13 |
6. Primary Endpoint
The registry lists the primary outcome as Median Time to Neutrophil Engraftment (Defined as an Absolute Neutrophil Count [ANC] Greater Than 500), measured over Statistic is calculated at Day 42 but ANC counts are measured daily up through discharge..
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | days |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is median time to neutrophil engraftment. Engraftment is defined as the first relevant milestone at which ANC is greater than 500. The registry states that ANC is measured daily up through discharge and that the statistic is calculated at Day 42. It identifies the full range as the measure of dispersion. The median and range themselves are not reported in the registry record.
In general, time to an event is useful when the timing of a clinical milestone matters, not just whether it has occurred by a particular visit. The median is the middle of the observed event-time distribution: it describes the point by which half the observations fall at or before that time, subject to how the analysis handles participants whose event time is not observed. The full range describes the lowest and highest recorded values, but does not show how observations are distributed between those limits.
Daily ANC measurements make it possible to identify when the specified count threshold is reached during the stated monitoring period. However, the registry record does not explain the operational rules for determining the event date, handling discharge, or dealing with participants who have no recorded engraftment by the analysis time. Those details matter to a time-to-event summary. Without the actual median, range, and analysis rules, the endpoint definition can be described, but the observed timing and its precision cannot be evaluated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clinically interpretable primary endpoint: median time to neutrophil engraftment, defined by ANC greater than 500. It also specifies daily ANC measurement up through discharge and a Day 42 calculation timeframe. However, the endpoint result is not reported in the registry record. There is therefore no posted median or full range available in the registry record for describing the observed timing or its spread.
The study is single-group and lists no comparator. Consequently, even if an outcome estimate were available, it would describe the participants treated in this study rather than a treatment contrast. A before-and-after or historical comparison cannot be assumed: none is specified in the registry record. The listed purpose was to determine whether adding parathyroid hormone would improve engraftment, but a stated purpose is not evidence that an improvement was observed.
The registry lists secondary outcomes covering graft-versus-host disease, platelet engraftment, transplant-related mortality, relapse, overall survival, and disease-free survival. Their results are not reported in the registry record. The record also gives no sponsor-reported hazard ratios or other effect estimates in the registry record for this analysis. It is not possible to assess the size, direction, or precision of effects for those outcomes.
Uncertainty cannot be quantified from an endpoint definition alone. A confidence interval would ordinarily communicate a range of values reasonably compatible with the data under an analysis model. No confidence interval for the primary endpoint is reported in the registry record. Nor does the record provide enough information to assess whether the observed participants represent a broader population of people receiving this transplant approach.
The enrollment is 13, and the record states that 13 patients received the transplant. This describes the recorded study size, not the precision of an unreported endpoint estimate. The study status is terminated, but the reason is not reported in the registry record. Termination alone does not reveal whether recruitment, safety, feasibility, or another issue affected the study.
The most defensible reading is therefore descriptive and limited: the registry records a single-group Phase II study designed to assess time to neutrophil engraftment following parathyroid hormone after transplantation, but the primary result and listed secondary outcome results are not reported in the registry record. No efficacy or causal conclusion follows from the design information alone.
9. Statistical Methods Explained
What does a median time measure?
In general, a time-to-event endpoint records how long it takes for a defined event to occur. Here, the event is neutrophil engraftment, defined as ANC greater than 500. The registry describes daily ANC measurement up through discharge and a median calculation at Day 42. A median is a location summary: it identifies the center of the observed event-time distribution. It is not the average time, and it does not show the whole distribution. The full range adds the smallest and largest values, but it can be strongly affected by extreme observations and does not indicate how densely the other observations cluster.
The registry record does not report the median or the range. It also does not describe how the analysis treated participants who were discharged before a relevant measurement, who did not reach the threshold during observation, or whose follow-up ended before engraftment was documented. In general, time-to-event analyses need explicit rules for such cases because the event time may be only partly observed. Those rules are not reported in the registry record, so no particular method should be assumed.
How should a single-group result be understood?
In general, a single-group study estimates or describes outcomes among participants who receive the intervention. Without a concurrent comparator, the observed endpoint cannot separate the intervention’s contribution from other influences, such as the underlying transplant context or participant characteristics. This is a design limitation, not a defect that can be repaired simply by calculating a more precise summary. The registry lists allocation as not applicable, masking as none, and the model as single group. It does not report a comparator arm.
A study purpose can frame the question, but does not establish the answer. The stated purpose was to determine whether adding parathyroid hormone after sequential cord blood transplantation would improve engraftment. The primary endpoint is related to that question, but the registry record does not report its numerical result. It is therefore not possible to say whether the observed time was shorter, longer, or otherwise different from any benchmark.
What would a confidence interval contribute?
In general, a confidence interval expresses statistical uncertainty around an estimate under the assumptions of the analysis. A narrow interval indicates more precise estimation than a wide interval, while neither interval width nor the estimate alone addresses all sources of bias. For this study, no confidence interval for the primary endpoint is reported in the registry record. Without an estimate and its uncertainty measure, precision cannot be judged from the endpoint description or the enrollment alone.
Time-to-event methods can also use hazard ratios when comparing event rates over time between groups. In general, a hazard ratio is a relative comparison of instantaneous event rates under a model; it is not the same as a ratio of median times. The record identifies no sponsor-reported hazard ratios in the registry record, and the single-group design gives no concurrent treatment contrast. No hazard ratio should be inferred.
What is the role of the measurement schedule?
The registry states that ANC counts are measured daily up through discharge, while the statistic is calculated at Day 42. These details describe the monitoring and stated timeframe, but they do not fully specify how the event time was assigned or how observations after discharge were handled. In general, the timing and completeness of measurements affect how accurately an event date can be located. The record does not provide additional operational definitions or follow-up procedures.
The primary endpoint lists the full range as dispersion. In general, dispersion describes variation across observations, and a range is easy to interpret because it marks the observed extremes. It is also an incomplete account of variability: it does not show the distribution’s shape or the uncertainty in the median. No range value is reported, and no other variability measure is listed in the registry record for this analysis.
What can be inferred from the study size?
The registry records enrollment of 13 and says 13 patients received the transplant. In general, the number of observed participants affects the amount of information available for estimating an outcome, but the actual precision also depends on the outcome distribution and analysis. The count by itself does not establish that an estimate is reliable or unreliable. The record does not report a sample-size justification, a target effect, or the assumptions used in planning. Any planning illustration based on an observed effect would not be the trial’s own design rationale or observed power.
The registry gives no analysis results for the primary endpoint in the registry record for this analysis. It also lists no analyses. That absence does not establish that no analyses were ever conducted; it means analysis details and results are not reported in the registry record. No model, adjustment strategy, or handling of repeated measurements should be inferred from the endpoint description alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists several secondary outcomes in addition to the primary endpoint. These include acute graft-versus-host disease grades II-IV at Day 100, chronic graft-versus-host disease, platelet engraftment, 100-day transplant-related mortality, relapse, overall survival, and disease-free survival. The registry record does not report results for these outcomes in the registry record for this analysis.
When a study evaluates multiple outcomes, general statistical practice may address multiplicity, because examining many hypotheses can increase the chance of at least one apparently notable result by chance. The registry record does not report whether any multiplicity adjustment was planned or used, how outcomes were prioritized beyond identifying the primary endpoint, or whether any interim analyses were conducted. No such procedures should be assumed.
The record does not describe missing-data rules, participant exclusions, or the analysis population used for each outcome. It records 13 enrolled participants and says 13 patients received the transplant, but that does not establish that every participant contributed data to every endpoint. For time-to-event outcomes, participants may have incomplete follow-up; for other outcomes, status may be unrecorded. In general, how incomplete observations are handled can affect estimates, but the method used in this study is not reported in the registry record.
The registry’s listed outcomes and study size alone cannot establish completeness of follow-up or the number analyzed for each endpoint. Without analysis populations, missing-data methods, and outcome results, readers cannot evaluate whether exclusions or incomplete observations could have influenced the reported findings. These are reporting gaps, not grounds for assuming a particular analysis choice.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Parathyroid Hormone (Teriparatide) | 8 | 13 | 61.5% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Parathyroid Hormone (Teriparatide) | 8 of 13 |
Most frequent serious adverse event terms
| Event term | Organ system | Parathyroid Hormone (Teriparatide) |
|---|---|---|
| Death | General disorders | 8 of 13 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Parathyroid Hormone (Teriparatide) |
|---|---|---|
| Death | General disorders | 8 of 13 |
Serious events with the highest arm-level rate: death (61.5%).
The registry’s adverse-event information lists 13 participants at risk in the parathyroid hormone group and 8 participants affected in the serious-event fields. It also lists 13 at risk and 8 affected in the other-event fields. The most frequent serious term shown is Death, with 61.5%. The registry record does not provide further detail in the registry record for this analysis about event timing, individual event descriptions, severity grading beyond the serious-event classification, or attribution to treatment.
These figures should be read as safety reporting, not as evidence that parathyroid hormone caused the events. In general, an adverse event occurring during a study is not necessarily caused by the intervention; causal attribution requires additional clinical and analytic information. The record does not specify whether the serious events were judged related to treatment. The deaths field is null, so it does not supply a separate death count in the registry entries. The listed Death term and percentage should not be converted into an unreported count or interpreted as a treatment-attributable mortality estimate.
The small recorded study group and lack of a control group also limit comparative safety interpretation. The registry’s safety figures describe the reported study group; they do not establish excess risk relative to another group. Further event details and attribution are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record documents a Phase II, single-group study of parathyroid hormone after sequential unrelated cord blood transplantation. It identifies median time to neutrophil engraftment as the primary endpoint, defines the threshold as ANC greater than 500, and states that ANC was measured daily up through discharge for a statistic calculated at Day 42. These details explain what the study set out to measure.
The primary endpoint result is not reported in the registry record. The registry therefore does not show the observed median time or full range, and it does not provide a confidence interval or another uncertainty measure for this endpoint in the registry record for this analysis. The secondary outcome results are also not reported in the registry record. The stated study purpose should not be treated as a finding.
The design does not include a comparator group. As a result, the record cannot establish what outcome would have occurred under a different treatment strategy, and it cannot isolate the effect of parathyroid hormone from other factors. A single-group observation can describe what was recorded among participants, but it does not on its own support a causal claim.
The safety listing includes serious-event information and identifies Death as a serious term, but the record does not provide treatment attribution in the registry record for this analysis. The status is terminated, and the reason for termination is not reported in the registry record. Neither the safety listing nor the status alone explains why the study ended.
In practical statistical terms, the available registry information supports interpretation of the question, design, endpoint definition, and some safety reporting. It does not support a conclusion about the magnitude or direction of an engraftment effect, the precision of an outcome estimate, comparative safety, or efficacy. Those conclusions require results and analysis details that are not reported in the registry record.
13. Why This Trial Matters Statistically
This study illustrates why an endpoint definition and an endpoint result are different kinds of information. The registry specifies a measurable milestone—neutrophil engraftment at ANC greater than 500—and describes daily measurement up through discharge. That level of definition helps identify what outcome was intended. But without the median and its full range, readers cannot learn the observed timing or how much it varied among participants.
The design also makes the distinction between description and causal inference especially important. The study is recorded as single group, with allocation not applicable and no masking. Its purpose concerns whether parathyroid hormone might improve engraftment, but the absence of a concurrent comparator means that the intervention’s contribution cannot be isolated from other influences. The study question and the design’s evidential limits must be read together.
The record is also a useful example of how registry status and safety reporting need context. The study is marked terminated, but the reason is not reported. The safety fields identify serious events, including Death, yet the available information does not establish treatment attribution. Neither fact should be over-interpreted in isolation.
More broadly, this case shows that statistical interpretation depends on transparent reporting of estimates, uncertainty, analysis populations, missing-data handling, and analysis methods, not only on a study’s objectives and listed outcomes. The registry record identifies the outcomes of interest, but the outcome results and many analysis details are not reported. Statistical restraint is therefore essential: the design can be characterized, while the study’s effect estimates cannot be evaluated from the available record.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The central limitation is that the primary endpoint’s numerical result is not reported in the registry record. Although the endpoint definition, timeframe, and full-range dispersion measure are described, the median and range values are absent. This prevents assessment of the observed timing and its variability. The listed secondary outcomes also have no result values in the registry record for this analysis.
The single-group design has no concurrent comparator. The record therefore cannot support a direct treatment contrast, and observed outcomes cannot be attributed to parathyroid hormone alone. The lack of masking is recorded, but the registry does not describe outcome-assessment procedures or whether any safeguards were used. The reason for termination is not reported, so its implications for recruitment, follow-up, or analysis cannot be determined.
The registry gives no detailed account of eligibility criteria, participant characteristics, treatment dose or duration, endpoint adjudication, analysis populations, missing-data handling, or statistical methods. It does not report a sample-size justification or interim-analysis plan. Without these details, it is difficult to assess how the observed group was selected, whether all participants contributed to each outcome, or how uncertainty was handled.
The safety information is limited in the registry entries. It identifies affected participants and a serious-event term, but does not report treatment attribution or event-level context. The listed safety figures cannot be interpreted as comparative risks because no control group is described. These gaps constrain both clinical interpretation and evaluation of the statistical evidence.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of Parathyroid Hormone Following Sequential Cord Blood Transplantation From an Unrelated Donor — NCT00393380.
- Registry record last updated 2013-04-29; status last verified 2013-04.
- Results first posted 2012-12-07.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT00393380 is a Phase II, single-group study of parathyroid hormone (teriparatide) after myeloablative sequential unrelated cord blood transplantation. The registry records 13 enrolled participants and says 13 patients received the transplant. The primary endpoint is median time to neutrophil engraftment, defined as ANC greater than 500; ANC was measured daily up through discharge, with the statistic calculated at Day 42 and full range listed as dispersion.
The primary endpoint result and the listed secondary outcome results are not reported in the registry record. The study is marked terminated, but the reason is not reported. The record identifies serious adverse-event information, including Death as a listed serious term, without reporting treatment attribution in the registry record for this analysis. The single-group design does not provide a concurrent control for causal or comparative interpretation.